United States: Intercept had tapped the accelerated approval in 2016 from the USFDA for its liver disease drug, based on which the staff reviewers said a confirmatory trial proved the drug to be ineffective on Wednesday.
In the accelerated pathway, there is provision for extra trial to prove the efficacy of the drug the companies want to market. For failure to do so, there is sometimes market withdrawal experienced by the companies.
As reported by Reuters, Ocaliva is Intercept’s oral drug which originally gained approval via the accelerated approval pathway for treating patients with rare ailments such as Primary biliary Cholangitis also known as PBC, this is a disease that leads to inflammation of small bile ducts in the liver and possibly the destruction of same.
The FDA in 2021 also wanted to localize the use of Ocaliva approved for PBC to those patients who had not been diagnosed with advanced cirrhosis or severe scarring of the liver.
The FDA had stated that liver deterioration which is life threatening occurs in some patient with advanced cirrhosis who where administered Ocaliva. The staff advisers stated on Wednesday that confirmatory trial of Ocaliva did not demonstrate that it was beneficial for PBC patients irrespective of advanced scarring.
They also questioned the drug’s safety as death or the need for liver transplantation in eleven patients who were on the drug during the clinical trial occurred whereas only two patients on placebo.
The concerns have been made prior to a meeting of independent experts on Friday in which the participants will make a vote on whether the drug has more benefits than risks or not. Intercept stated that only one of those cases could have been a result of the drug with the remainder citing other cause such as liver transplant.
Its U. S. commercial rights are owned by the firm Kf which was bought by Italy’s Alfasigma for about $800 million last year. Intercept had made the first half sales sales of $152 million in the first half of the 2023.
Last month, the FDA approved Gilead’s (GILD.O) Livdelzi, an accelerated treatment option for PBC.
The process of accelerated approvals enables the FDA to bring new drugs to the people faster when the drug is needed to treat fatal diseases; however, they have been criticized because some of the advanced drugs are not really effective as thought initially.
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